Country for PR: United States
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Friday, December 25 2020 - 02:19
INOVIO Announces Publication of Phase 1 Data from its COVID-19 DNA Vaccine Candidate, INO-4800 in The Lancet's EClinicalMedicine
PLYMOUTH MEETING, Pa., Dec. 24, 2020 /PRNewswire-AsiaNet/ --

-- Peer-reviewed Phase 1 data shows INO-4800 to be immunogenic in 100% of 
subjects, inducing neutralizing antibody and/or T cell responses

-- INO-4800 demonstrates favorable safety and tolerability, with no serious 
adverse events reported

-- Offers best-in-class thermostability, including a five-year projected shelf 
life at normal refrigeration temperature and no frozen transport or storage 

INOVIO (NASDAQ:INO), a biotechnology company focused on bringing to market 
precisely designed DNA medicines to treat and protect people from infectious 
diseases and cancer, today announced the publication of peer-reviewed Phase 1 
clinical data from the first cohort of 40 participants for its COVID-19 DNA 
vaccine candidate, INO-4800, in EClinicalMedicine, an open access clinical 
journal published by The Lancet. 

The paper, titled "Safety and immunogenicity of INO-4800 DNA vaccine against 
SARS-CoV-2: a preliminary report of an open-label, Phase 1 clinical trial," 
found that INO-4800 was immunogenic in all vaccinated subjects, effectively 
generating an immune response of humoral (including neutralizing antibodies) 
and/or cellular responses (both CD4 and CD8 T cells). 

Additionally, Phase 1 clinical data found INO-4800 to have a favorable safety 
and tolerability profile with no serious adverse events reported; only six 
Grade 1 adverse events (AEs) were observed, primarily minor injection site 
reactions. Notably, these only occurred on the day of the first or second 
dosing, and the AEs did not increase in frequency with the second 

INO-4800, beyond being safe and tolerable, is stable at room temperature for 
more than a year, at 37o C (98.6o F) for more than a month, has a five-year 
projected shelf life at normal refrigeration temperature [i.e., at 2-8o C / 
35.6 – 46.4o F] and does not need to be frozen during transport or storage – 
all critical factors for timely global distribution in the fight against 

Dr. J. Joseph Kim, President and CEO of INOVIO, said, "We are very pleased to 
share peer-reviewed Phase 1 clinical data for INO-4800 published in The 
Lancet's EClinicalMedicine, and are grateful for the support of all 
participants and investigator staff involved in the clinical trial."

Dr. Stanley Plotkin, Professor Emeritus at The Wistar Institute, said, 
"INOVIO's DNA vaccine appeared to be quite safe with few significant reactions 
but yet induced both antibody and T cell responses to SARS-CoV-2." 

Findings from the Phase 1 Clinical Trial

   -- The Phase 1 clinical trial of INO-4800 initially enrolled 40 healthy
      adult volunteers, ages 18 to 50, at two U.S. sites with funding from the
      Coalition for Epidemic Preparedness Innovations (CEPI). 
   -- The participants were enrolled into 1.0 mg and 2.0 mg dose cohorts; each
      participant received two doses of INO-4800 four weeks apart. Each dose
      was administered by intradermal injection using INOVIO's proprietary
      smart device CELLECTRA(R). 
   -- Thirty-nine subjects completed both doses. One subject in the 2.0 mg
      group discontinued trial participation prior to receiving the second dose
      due to lack of transportation to the clinical site; discontinuation was
      unrelated to the study or the dosing. One subject was deemed to be
      seropositive at trial entry. 
   -- The 1.0 mg and 2.0 mg dose group both demonstrated seroconversion in 95%
      of the subjects, respectively, with 78% demonstrating neutralizing
      antibodies in the 1.0 mg dose group and 84% demonstrating neutralizing
      antibodies in the 2.0 mg dose group. 
   -- Cellular (T cell) response were observed to multiple regions of the spike
      protein including the RBD region. 74% had measurable cellular responses
      at the 1.0 mg dose group and 100% of the subjects in the 2.0 mg dose
      group demonstrated cellular responses. 
   -- Through week 8, no serious adverse events were reported. Only 6 related
      Grade 1 adverse events in 5 subjects were observed, primarily mild
      injection site reactions (e.g., redness); none of these increased in
      frequency with the second administration. 
   -- All 38 subjects who were evaluable for immunogenicity had balanced
      cellular and humoral immune responses following the second dose of INO
   -- identifier: NCT04336410.

INOVIO is currently conducting the Phase 2 segment of its planned Phase 2/3 
clinical trial for INO-4800, called INNOVATE (INOVIO INO-4800 Vaccine Trial for 
Efficacy). INNOVATE is a randomized, blinded, placebo-controlled safety and 
efficacy trial of INO-4800 to be conducted in adults in the U.S. It will be 
funded by the U.S. Department of Defense (DoD) Joint Program Executive Office 
for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) in 
coordination with the Office of the Assistant Secretary of Defense for Health 
Affairs (OASD(HA)) and the Defense Health Agency (DHA).

The DoD has agreed to provide funding for both the Phase 2 and Phase 3 segments 
of the INNOVATE clinical trial, in addition to the $71 million of funding 
previously announced in June 2020 for the large-scale manufacture of the 
company's proprietary smart device CELLECTRA(R) 3PSP and the procurement of 
CELLECTRA(R) 2000 devices. 

INOVIO also recently announced ( 
) the first dosing of its first subject in its Phase 2 clinical trial for 
INO-4800 in China, in collaboration with Advaccine. The company is currently in 
Phase 1/2a trials for INO-4800 in South Korea in partnership with The 
International Vaccine Institute and the Korea National Institute for Health.

About the INO-4800 "INNOVATE" Phase 2/3 Clinical Trial 

The lead Principal Investigator for the INNOVATE trial is Dr. Pablo Tebas, 
Professor of Medicine at the Hospital of the University of Pennsylvania. The 
Phase 2 segment of the clinical trial is designed to evaluate safety, 
tolerability and immunogenicity of INO-4800 in a 2-dose regimen (1.0 mg or 2.0 
mg), in a three-to-one randomization to receive either INO-4800 or placebo for 
each dose, to confirm the more appropriate dose(s) for each of three age groups 
(18-50 years, 51-64 years and 65 years and older) for the subsequent Phase 3 
efficacy evaluation. The company intends to work diligently to ensure diversity 
in enrollment, targeting specific populations that are working or residing in 
environments with high infection rates and/or areas where there is greater risk 
of exposure to SARS-CoV-2, for whom exposure may be relatively prolonged or for 
whom personal protective equipment (PPE) may be inconsistently used, especially 
in confined settings.

In the Phase 3 segment of the clinical trial, INOVIO intends to enroll healthy 
men and non-pregnant women 18 years and older, to evaluate the efficacy of the 
proposed dose(s) based on the data from the Phase 2 evaluation. Participants 
will be enrolled in a one-to-one randomization to receive either INO-4800 or a 
placebo. The Phase 3 segment will be case-driven with the final number of 
enrollees to be determined by the incidence of COVID-19 during the Phase 3 
segment. The primary endpoint of the Phase 3 segment will be virologically 
confirmed COVID-19 disease.

About INOVIO's Global Coalition Advancing INO-4800

INOVIO has assembled a global coalition of collaborators, partners and funders 
to rapidly advance the development of INO-4800. R&D collaborators to date 
include The Wistar Institute, the University of Pennsylvania, the University of 
Texas, Fudan University and Laval University. INOVIO has partnered with 
Advaccine and the International Vaccine Institute to conduct clinical trials of 
INO-4800 in China and South Korea, respectively. INOVIO is also assessing 
nonclinical efficacy of INO-4800 in several animal challenge models with Public 
Health England (PHE) and Commonwealth Scientific and Industrial Research 
Organization (CSIRO) in Australia. INOVIO is working with a team of contract 
manufacturers including Kaneka Eurogentec, Thermo Fisher Scientific, 
Richter-Helm BioLogics, and Ology Bioservices to manufacture INO-4800 on a 
commercial scale and is seeking additional external funding and partnerships to 
further scale up manufacturing capacities to satisfy the urgent global demand 
for safe and effective vaccines. To date, the Coalition for Epidemic 
Preparedness Innovations (CEPI), the Bill & Melinda Gates Foundation, and the 
U.S. Department of Defense have contributed significant funding to the 
advancement and manufacturing of INO-4800.

About INO-4800

INO-4800 is INOVIO's DNA vaccine candidate against SARS-CoV-2, the novel 
coronavirus that causes COVID-19. INOVIO has extensive experience working with 
coronaviruses and was the first company to initiate a Phase 2a trial for 
INO-4700, a DNA vaccine candidate for a related coronavirus that causes Middle 
East Respiratory Syndrome (MERS).

Composed of an optimized DNA plasmid, INO-4800 is delivered directly into cells 
in the body via a proprietary smart device to produce a robust, safe and 
tolerable immune response. INO-4800 is the only nucleic-acid based vaccine that 
is stable at room temperature for more than a year, at 37o C for more than a 
month, has a five-year projected shelf life and does not need to be frozen in 
transport of storage, which are important factors when implementing mass 

About INOVIO's DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused 
on HPV-associated diseases, cancer, and infectious diseases, including 
coronaviruses associated with MERS and COVID-19, being developed under grants 
from the Coalition for Epidemic Preparedness Innovations (CEPI) and the U.S. 
Department of Defense. DNA medicines are composed of optimized DNA plasmids, 
which are small circles of double-stranded DNA that are synthesized or 
reorganized by a computer sequencing technology and designed to produce a 
specific immune response in the body.

INOVIO's DNA medicines deliver optimized plasmids directly into cells 
intramuscularly or intradermally using INOVIO's proprietary hand-held smart 
device called CELLECTRA(R). The CELLECTRA(R) device uses a brief electrical 
pulse to reversibly open small pores in the cell to allow the plasmids to 
enter, overcoming a key limitation of other DNA and other nucleic acid 
approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the 
cell to produce the targeted antigen. The antigen is processed naturally in the 
cell and triggers the desired T cell and antibody-mediated immune responses. 
Administration with the CELLECTRA(R) device ensures that the DNA medicine is 
efficiently delivered directly into the body's cells, where it can go to work 
to drive an immune response. INOVIO's DNA medicines do not interfere with or 
change in any way an individual's own DNA. The advantages of INOVIO's DNA 
medicine platform are how fast DNA medicines can be designed and manufactured; 
the stability of the products, which do not require freezing in storage and 
transport; and the robust immune response, safety profile, and tolerability 
that have been observed in clinical trials.

With more than 2,000 patients receiving INOVIO investigational DNA medicines in 
more than 7,000 applications across a range of clinical trials, INOVIO has a 
strong track record of rapidly generating DNA medicine candidates with 
potential to meet urgent global health needs.


INOVIO is a biotechnology company focused on rapidly bringing to market 
precisely designed DNA medicines to treat and protect people from infectious 
diseases, cancer, and diseases associated with HPV. INOVIO is the first and 
only company to have clinically demonstrated that a DNA medicine can be 
delivered directly into cells in the body via a proprietary smart device to 
produce a robust and tolerable immune response. Specifically, INOVIO's lead 
candidate VGX-3100, currently in Phase 3 trials for precancerous cervical 
dysplasia, destroyed and cleared high-risk HPV 16 and 18 in a Phase 2b clinical 
trial. High-risk HPV is responsible for 70% of cervical cancer, 91% of anal 
cancer, and 69% of vulvar cancer. Also in development are programs targeting 
HPV-related cancers and a rare HPV-related disease, recurrent respiratory 
papillomatosis (RRP); non-HPV-related cancers glioblastoma multiforme (GBM) and 
prostate cancer; as well as externally funded infectious disease DNA vaccine 
development programs in Zika, Lassa fever, Ebola, HIV, and coronaviruses 
associated with MERS and COVID-19 diseases. Partners and collaborators include 
Advaccine, ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates 
Foundation, Coalition for Epidemic Preparedness Innovations (CEPI), Defense 
Advanced Research Projects Agency (DARPA)/Joint Program Executive Office for 
Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND)/Department 
of Defense (DoD), HIV Vaccines Trial Network, International Vaccine Institute 
(IVI), Kaneka Eurogentec, Medical CBRN Defense Consortium (MCDC), National 
Cancer Institute, National Institutes of Health, National Institute of Allergy 
and Infectious Diseases, Ology Bioservices, the Parker Institute for Cancer 
Immunotherapy, Plumbline Life Sciences, Regeneron, Richter-Helm BioLogics, 
Thermo Fisher Scientific, University of Pennsylvania, Walter Reed Army 
Institute of Research, and The Wistar Institute. INOVIO also is a proud 
recipient of 2020 Women on Boards "W" designation recognizing companies with 
more than 20% women on their board of directors. For more information, visit


Media: Jeff Richardson, +1-267-440-4211, 
Investors: Ben Matone, +1-484-362-0076,

This press release contains certain forward-looking statements relating to our 
business, including our plans to develop and manufacture DNA medicines, our 
expectations regarding our research and development programs, including the 
conduct of the Phase 2/3 clinical trial of INO-4800, and our ability to 
successfully manufacture and produce large quantities of our product candidates 
if they receive regulatory approval. Actual events or results may differ from 
the expectations set forth herein as a result of a number of factors, including 
uncertainties inherent in preclinical studies, clinical trials, product 
development programs and commercialization activities and outcomes, our ability 
to secure sufficient manufacturing capacity to mass produce our product 
candidates, the availability of funding to support continuing research and 
studies in an effort to prove safety and efficacy of electroporation technology 
as a delivery mechanism or develop viable DNA medicines, our ability to support 
our pipeline of DNA medicine products, the ability of our collaborators to 
attain development and commercial milestones for products we license and 
product sales that will enable us to receive future payments and royalties, the 
adequacy of our capital resources, the availability or potential availability 
of alternative therapies or treatments for the conditions targeted by us or our 
collaborators, including alternatives that may be more efficacious or cost 
effective than any therapy or treatment that we and our collaborators hope to 
develop, issues involving product liability, issues involving patents and 
whether they or licenses to them will provide us with meaningful protection 
from others using the covered technologies, whether such proprietary rights are 
enforceable or defensible or infringe or allegedly infringe on rights of others 
or can withstand claims of invalidity and whether we can finance or devote 
other significant resources that may be necessary to prosecute, protect or 
defend them, the level of corporate expenditures, assessments of our technology 
by potential corporate or other partners or collaborators, capital market 
conditions, the impact of government healthcare proposals and other factors set 
forth in our Annual Report on Form 10-K for the year ended December 31, 2019, 
our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 and 
other filings we make from time to time with the Securities and Exchange 
Commission. There can be no assurance that any product candidate in our 
pipeline will be successfully developed, manufactured or commercialized, that 
final results of clinical trials will be supportive of regulatory approvals 
required to market products, or that any of the forward-looking information 
provided herein will be proven accurate. Forward-looking statements speak only 
as of the date of this release, and we undertake no obligation to update or 
revise these statements, except as may be required by law.

SOURCE: INOVIO Pharmaceuticals, Inc.